Vaccinex, Inc. Receives Orphan Drug Designation from U.S. FDA for its Lead Antibody Product Candidate, VX15, as a Potential Treatment for Huntington’s Disease

VX15 for the potential treatment of HD also received Fast Track status from FDA in July 2016

ROCHESTER, N.Y., August 23, 2016 (GLOBE NEWSWIRE) – Vaccinex, Inc., a clinical-stage biotechnology company engaged in the discovery and development of therapeutic monoclonal antibodies to treat patients with cancer and neurodegenerative diseases, today announced that its lead antibody product candidate, VX15, which is currently being investigated in a Phase 2 clinical trial for the treatment of Huntington’s disease (the “SIGNAL” trial), has received Orphan Drug Designation by the U.S. Food and Drug Administration (“FDA”).

The SIGNAL trial is based on the company’s prior research of neurodegenerative disease mechanisms, where it was demonstrated in preclinical models that semaphorin 4D (“SEMA4D”) triggers activation of both microglia and astrocytes, the innate inflammatory cells of the central nervous system. The chronic activation of microglia and astrocytes has been implicated as a potentially important disease mechanism in HD, progressive multiple sclerosis (“MS”) and other neurodegenerative disorders. The VX15 antibody is designed to block the functional activity of SEMA4D. The SIGNAL clinical trial builds upon preclinical studies in an animal model of HD and safety data from a Phase 1 dose-escalation clinical trial of VX15 in MS patients that was completed in November 2014.

“We are pleased to receive Orphan Drug Designation from the FDA for VX15 as a potential treatment for Huntington’s disease. SIGNAL is an important proof of concept study for the role of inflammation and, in particular, SEMA4D in neurodegenerative processes. We hope that this study will contribute to treatment of HD, a serious disease for which there are, as yet, no approved disease-modifying therapies,” said Maurice Zauderer, Ph.D., Chief Executive Officer of Vaccinex.

The Orphan Drug Act provides for economic incentives to encourage the development of drugs intended to treat, diagnose or prevent rare diseases and conditions affecting (i) fewer than 200,000 people in the United States or (ii) more than 200,000 people in the United States but for which there is no reasonable expectation for recouping development and commercialization costs based on U.S. sales. Orphan drug designation would provide seven years of market exclusivity in the United States, with certain exceptions, if market approval is granted for VX15. Additional incentives may include tax credits related to clinical trial expenses and waivers from FDA user fees.